Biostats Flashcards
Adjusted Rate (adjustment)
a summarizing procedure for a statistical measure in which the effects of differences in composition of the populations being compare have been minimized by statistical methods. Often performed on rates or relative risk, commonly because of differing age distributions in populations that are being compared. The mathematical procedure commonly used to adjust rates for age differences is direct or indirect standardization. Example: adjustment by regression analysis and by standardization.
Alpha (α)
the probability of a type I error, the error of rejecting a true null hypothesis (declaring a difference exists when it does not)
Alternative Hypothesis
- A supposition, arrived at from observation or reflection, that leads to refutable predictions. 2. Any conjecture cast in a form that will allow it to be tested and refuted.
Analysis of Variance (ANOVA)
The separation of variance attributable to one cause from the variance attributable to others. By partitioning the total variance of a set of observations into parts due to particular factors, for example, sex, treatment group, etc, and comparing variances (mean squares) by way of F-tests, differences between means can be assessed. The simplest analysis of this type involves a one-way design, in which N subjects are allocated, usually at random, to the k different levels of a single factor. The total variation in the observations is then divided into a part due to differences between level means (the between groups sum of squares) and a part due to the differences between subjects in the same group (the within groups sum of squares, also known as the residual sum of squares). These terms are usually arranged as an analysis of variance table.
If the means of the populations represented by the factor levels are the same, then within the limits of random variations, the between groups mean square and within groups mean square, should be the same. Whether this is so can, if certain assumptions are met, be assessed by a suitable F-test are that the response variable is normally distributed in each population and that the populations have the same variance. Essentially an example of a generalized linear model with an identity link function and normally distributed errors.
Bayes’ theorem
A procedure for revising and updating the probability of some event in the light of new evidence. The theorem originates in an essay by the Reverend Thomas Bayes. In its simplest form the theorem may be written in terms of conditional probabilities as,
pr (Bj| A)=
where Pr( A | Bj ) denotes the conditional probability of event A conditional on event Bj and B1 , B2 ,…,Bk are mutually exclusive and exhaustive events. The theorem gives the probabilities of the Bj when A is known to have occurred. The quantity Pr( Bj ) is termed the prior probability and Pr( Bj | A ) the posterior probability . Pr( A | Bj ) is equivalent to the (normalized) likelihood , so that the theorem may be restated as posterior (prior) x (likelihood).
Beta (β)
The probability of a type II error, the error of failing to reject a false null hypothesis, i.e. declaring that a difference does not exist when in fact it does.
Bias
In general terms, deviations of results or inferences from the truth, or processes leading to such deviation. More specifically, the extent to which the statistical method used in a study does not estimate the quantity thought to be estimated, or does not test the hypothesis to be tested. In estimated usually measured by the difference between a parameter estimate and its expected value. An estimator for which is said to be unbiased .
Binary Variable (Binary Observation)
Observations which occur in one of two possible states, these often being labeled 0 and I. Such data is frequently encountered in medical investigations; commonly occurring examples include ‘dead/alive’, ‘improved/not improved’ and ‘depressed/not depressed.’ Data involving this type of variable often require specialized techniques for their analysis such as logical regression.
Binomial Distribution
The distribution of the number of ‘successes’, X, in a series of n- independent Bernoulli trials where the probability of success at each trial is p and the probability of failure is q = 1- p . Specifically the distribution is given by
Pr(X=x) = n!/x!(n-x)![q^(n-x)], x = 0, 1, 2 ……, n
The mean, variance, skewness and kurtosis of the distribution are as follows: mean = np variance = npq skewness = ( q - p )/( npq ) 1/2 kurtosis = 3-(6/n)+1/npq
Biostatistics
A branch of science which applies statistical methods to biological problems. The science of biostatistics encompasses the design of biological experiments, especially in medicine and health sciences.
Bivariate
outcomes belong to two categories, e.g. yes/no, acceptable/defective “bivariate binomial distribution”.
Blinded Study (Blinding)
A procedure used in clinical trials to avoid the possible bias that might be introduced if the patient and/or doctor knew which treatment the patient is receiving. If neither the patient nor doctor are aware of which treatment has been given the trial is termed double-blind. If only one of the patient or doctor is unaware, the trial is called single-blind. Clinical trials should use the maximum degree of blindness that is possible, although in some areas, for example, surgery, it is often impossible for an investigation to be double-blind.
Bonferroni correction
A procedure for guarding against an increase in the probability of a type I error when performing multiple significance tests. To maintain the probability of a type I error at some selected value (α), each of the m tests to be performed is judged against a significance level (α/m ). For a small number of simultaneous tests (up to five) this method provides a simple and acceptable answer to the problem of multiple testing. It is however highly conservative and not recommended if large numbers of tests are to be applied, when one of the many other multiple comparison procedures available is generally preferable.
Case-Control Study
(Syn: case comparison study, case compeer study, case history study, case referent study, retrospective study) The observational epidemiologic study of persons with the disease (or other outcome variable) of interest and a suitable control (comparison, reference) group of persons without the disease.
Categorical Data
Categorical data represent types of data which may be divided into groups. Examples of categorical variables are race, sex, age group, and educational level. While the latter two variables may also be considered in a numerical manner by using exact values for age and highest grade completed, it is often more informative to categorize such variables into a relatively small number of groups.
Censored Information
An observation (Xi) on some variable of interest is said to be censored if it is known only that Xi =Li ( left-censored) or Xi =Ui ( right-censored) where Li and Ui are fixed values. Such observations arise most frequently in studies where the main purpose variable is time until a particular event occurs (for example, time to death) when at the completion of the study, the event of interest has not happened to a number of subjects.
Central Limit Theorem
If a random variable Y has population mean µ and population variance σ2, then the sample mean, , based on n observations, has an appropriate normal distribution with a mean µ and variance σ2/ n , for sufficiently large n. The theorem occupies an important place in statistical theory. In short, the Central Limit Theorem states that if the sample size is large enough, the distribution of sample means can be approximated by a normal distribution, even if the original population is not normally distributed.
Chi-Square Distribution
The Chi-Square distribution is based on a normally distributed population with variance σ2, with randomly selected independent samples of size n and computed sample variance s2 for each sample. The sample statistic X2= ( n – 1) s2/σ2. The chi-square distribution is skewed, the values can be zero or positive but not negative, and it is different for each number of degrees of freedom. Generally, as the number of degrees of freedom increases, the chi-square distribution approaches a normal distribution.
Chi-square statistic
A statistic having, at least approximately, a chi-squared distribution.
Chi-square test for trend
A test applied to a two-dimensional contingency table in which one variable has two categories and the other has k ordered categories, to assess whether there is a difference in the trend of the proportions in the two groups. The result of using the ordering in this way is a test that is more powerful than using the chi-squared statistic to test for independence.
clinical trial (phases 1-4)
Syn: therapeutic trial) A research activity that involves the administration of a test regimen to humans to evaluate its efficacy and safety. The term is subject to wide variation in usage, from the first use in humans without any control treatment to a rigorously designed and executed experiment involving test and control treatments and randomization. Several phases of clinical trials are distinguished:
Phase I trial Safety and pharmacologic profiles. The first introduction of a candidate vaccine or a drug into a human population to determine its safety and mode of action. In drug trials, this phase may include studies of dose and route of administration. Phase I trials usually involve fewer than 100 healthy volunteers.
Phase II trial Pilot efficacy studies. Initial trial to examine efficacy usually in 200 to 500 volunteers; with vaccines, the focus is on immunogenicity, and with drugs, on demonstration of safety and efficacy in comparison to other existing regimens. Usually but not always, subjects are randomly allocated to study and control groups.
Phase III trial Extensive clinical trial. This phase is intended for complete assessment of safety and efficacy. It involves larger numbers, perhaps thousands, of volunteers, usually with random allocation to study and control groups, and may be a multicenter trial.
Phase IV trial With drugs, this phase is conducted after the national drug registration authority (e.g., the Food and Drug Administration in the United States) has approved the drug for distribution or marketing. Phase IV trials may include research designed to explore a specific pharmacologic effect, to establish the incident of adverse reactions, or to determine the effects of long-term use. Ethical review is required for phase IV clinical trials, but not for routine post marketing surveillance.
coefficient of variation (CV)
he measure of spread for a set of data defined as
100 x standard deviation / mean
CV = s/x bar(100) = sample
CV = σ/µ(100) = population
Originally proposed as a way of comparing the variability in different distributions, but found to be sensitive to errors in the mean. Simpler definition: The ratio of the standard deviation to the mean. This is meaningful only if the variable is measured on a ratio scale.
Cohort Study
(Syn: concurrent, follow-up, incidence, longitudinal, prospective study) The analytic method of epidemiologic study in which subsets of a defined population can be identified who are, have been, or in the future may be exposed or not exposed, or exposed in different degrees, to a factor or factors hypothesized to influence the probability of occurrence of a given disease or other outcome.
complementary event
Mutually exclusive events A and B for which Pr(A) + Pr(B) = 1
where Pr denotes probability.
conditional probability
The probability that an event occurs given the outcome of some other event. Usually written, Pr(A l B). For example, the probability of a person being colour blind given that the person is male is about 0.1, and the corresponding probability given that the person is female is approximately 0.0001. It is not, of course, necessary that Pr(A l B) = Pr(A l B); the probability of having spots given that a patient has measles, for example, is very high, the probability of measles given that a patient has spots is, however, much less. If Pr(A l B) = Pr(A l B) then the events A and B are said to be independent.
confidence interval (CI)
A range of values, calculated from the sample observations, that is believed, with a particular probability, to contain the true value of a population parameter. A 95% confidence interval, for example, implies that were the estimation process repeated again and again, then 95% of the calculated intervals would be expected to contain the true parameter value. Note that the stated probability level refers to properties of the interval and not to the parameter itself which is not considered a random variable.
confounding variable
an extraneous variable in a statistical model that correlates (positively or negatively) with both the dependent variable and the independent variable . The methodologies of scientific studies therefore need to control for these factors to avoid what is known as a type 1 error : A ‘false positive’ conclusion that the dependent variables are in a causal relationship with the independent variable . Such a relation between two observed variables is termed a spurious relationship . Thus, confounding is a major threat to the validity of inferences made about cause and effect, i.e. internal validity , as the observed effects should be attributed to the confounder rather than the independent variable. By definition, a confounding variable is associated with both the probable cause and the outcome. The confounder is not allowed to lie in the causal pathway between the cause and the outcome: If A is thought to be the cause of disease C, the confounding variable B may not be solely caused by behaviour A; and behaviour B shall not always lead to behaviour C. An example: Being female does not always lead to smoking tobacco, and smoking tobacco does not always lead to cancer. Therefore, in any study that tries to elucidate the relation between being female and cancer should take smoking into account as a possible confounder. In addition, a confounder is always a risk factor that has a different prevalence in two risk groups (e.g. females/males). (Hennekens, Buring & Mayrent, 1987).
contingency table (two-way frequency table)
The table arising when observations on a number of categorical variables are cross-classified. Entries in each cell are the number of individuals with the corresponding combination of variable values. Most common are two-dimensional tables involving two categorical variables.
The analysis of such two-dimensional tables generally involves testing for the independence of the two variables using the familiar chi-squared statistics. Three- and higher-dimensional tables are now routinely analyzed using log-linear models.
continuous data
result from infinitely many possible values that correspond to some continuous scale that covers a range of values without gaps, interruptions or jumps, e.g. blood pressure.
controlled trial
A Phase III clinical trial in which an experimental treatment is compared with a control treatment, the latter being either the current standard treatment or a placebo.
correlation coefficient r (Pearson product moment)
An index that quantifies the linear relationship between a pair of variables. In a bivariate normal distribution, for example, the parameter, p. An estimator of p obtained from n sample values of the two variables of interest, (x1, y1), (x2, y2),…,(xn,yn), is Pearson’s product moment correlation coefficient, r, given by
The coefficient takes values between -1 and 1, with the sign indicating the direction of the relationship and the numerical magnitude its strength. Values of -1 and 1 indicate that the sample values fall on a straight line. A value of zero indicates the lack of any linear relationship between the two variables.
covariate
Often used simply as an alternative name for explanatory variables, but perhaps more specifically to refer to variables that are not of primary interest in an investigation, but are measured because it is believed that they are likely to affect the response variable and consequently need to be included in analyses and model building.
cox regression model (Proportional Hazards Model)
A statistical model used in survival analysis developed by D.R. Cox in 1972 asserting that the effect of the study factors on the hazard rate in the study population is multiplicative and does not change over time.
critical value
The value with which a statistic calculated from sample data is compared in order to decide whether a null hypothesis should be rejected. The value is related to the particular significance level chosen.
crossover rate
The proportion of patients in a clinical trial transferring from the treatment decided by an initial random allocation to an alternative one.
cross-sectional study
(Syn: disease frequency survey, prevalence study) A study that examines the relationship between diseases (or other health-related characteristics) and other variables of interest as they exist in defined population at one particular time.
cumulative frequency distribution
The tabulation of a sample of observations in terms of numbers falling below particular values. The empirical equivalent of the cumulative probability distribution. An example of such a tabulation is shown below.
degrees of freedom
An elusive concept that occurs throughout statistics. Essentially the term means the number of independent units of information in a sample relevant to the estimation of a parameter or calculation of a statistic. For example, in a two-by-two contingency table with a given set of marginal totals, only one of the four cell frequencies is free and the table has therefore a single degree of freedom. In many cases the term corresponds to the number of parameters in a model. Also used to refer to a parameter of various families of distributions, for example, Student’s t-distribution and the F-distribution.
dependent variable (response or outcome variable)
The variable of primary importance in investigations since the major objective is usually to study the effects of treatment and/or other explanatory variables on this variable and to provide suitable models for the relationship between it and the explanatory variables.
descriptive statistics
A general term for methods of summarizing and tabulating data that make their main features more transparent. For example, calculating means and variances and plotting histograms.
dichotomous observation
A nominal measure with two outcomes (examples are gender male or female; survival yes or no); also called binary. See dichotomous data.
dichotomous scale
one that arranges items into either of two mutually exclusive categories, e.g. yes/no, alive/dead.
discrete data
result when the number of possible values is either a finite number or a “countable” number.
Discrete variable
a countable and finite variable, for example grade:
1, 2, 3, 4…- 12.
distribution (population)
In statistics this term is used for any finite or infinite collection of ‘units’, which are often people but may be, for example, institutions, events, etc.
double-blinded trial
A procedure used in clinical trials to avoid the possible bias that might be introduced if the patient and/or doctor knew which treatment the patient is receiving. If neither the patient nor doctor are aware of which treatment has been given the trial is termed double-blind.
dummy coding
Dummy coding provides one way of using categorical predictor variables in various kinds of estimation models (see also effect coding), such as, linear regression. Dummy coding uses only ones and zeros to convey all of the necessary information on group membership.
http://www.ats.ucla.edu/stat/mult_pkg/faq/general/dummy.htm
dummy variable (indicator variable)
in statistics, a variable taking only one of two possible values, one (usually 1) indicating the presence of a condition, and the other (usually 0) indicating the absence of the condition, used mainly in regression analysis.
effect or effect size
a measure of the strength of the relationship between two variables. In scientific experiments, it is often useful to know not only whether an experiment has a statistically significant effect, but also the size of any observed effects. In practical situations, effect sizes are helpful for making decisions. Effect size measures are the common currency of meta-analysis studies that summarize the findings from a specific area of research.
Effective sample size
The sample size after dropouts, deaths and other specified exclusions from the original sample.
expected frequencies
A term usually encountered in the analysis of contingency tables. Such frequencies are estimates of the values to be expected under the hypothesis of interest. In a two-dimensional table, for example, the values under independence are calculated from the product of the appropriate row and column totals divided by the total number of observations.
experiment (in probability)
A probability experiment involves performing a number of trials to measure the chance of the occurrence of an event our outcome.
http://www.uic.edu/classes/upp/upp503/sanders4-5.pdf
experiment
study in which the investigator intentionally alters one or more factors under controlled conditions in order to study the effects of doing so.
explanatory variable
The variables appearing on the right-hand size of the equations defining, for example, multiple regression or logistic regression, and which seek to predict or ‘explain’ the response variable. Also commonly known as the independent variables, although this is not to be recommended since they are rarely independent of one another.
factor
An event, characteristic, or other definable entity that brings about a change in a health condition or other defined outcome.
factor analysis
A set of statistical methods for analyzing the correlations among several variables in order to estimate the number of fundamental dimensions that underlie the observed data and to describe and measure those dimensions. Used frequently in the development of scoring systems for rating scales and questionnaires.
factorial designs
Designs which allow two or more questions to be addressed in an investigation. The simplest factorial design is one in which each of two treatments or interventions are either present or absent, so that subjects are divided into four groups; those receiving neither treatment, those having only the first treatment, those having only the second treatment and those receiving both treatments. Such designs enable possible interactions between factors to be investigated. A very important special case of a factorial design is that where each of k factors of interest has only two levels; these are usually known as 2kfactorial designs. A single replicate of a 2kdesign is sometimes called an unreplicated factorial.
false-negative
The proportion of cases in which a diagnostic test indicates disease is absent in patients who have the disease.
v
false-positive
The proportion of cases in which a diagnostic test indicates disease is present in disease-free patients.
F distribution (variance ratio distribution)
The distribution of the ratio of two independent quantities each of which is distributed like a variance in normally distributed samples. So named in honor of R.A. Fisher who first described the distribution.
Fisher’s exact test
An alternative procedure to use of the chi-squared statistic for assessing the independence of two variables forming a two-by-two contingency table particularly when the expected frequencies are small. The method consists of evaluating the sum of the probabilities associated with the observed table and all possible two-by-two tables that have the same row and column totals as the observed data but exhibit more extreme departure from independence. The probability of each table is calculated from the hypergeometric distribution.
Fisher’s z-transformation
A transformation of Pearson’s product moment correlation coefficient, r, given by
z = 1/2 1n[(1+r)/(1-r)]
The statistic z has a normal distribution with mean
1/2 1n[(1+p)/(1-p)]
where ? is the population correlation value and variance 1/( n -3) where n is the sample size. The transformation may be used to test hypotheses and to contrast confidence intervals for ?.
frequency (occurrence)
a general term describing the frequency or occurrence of a disease or other attribute or event in a population without distinguishing between incidence and prevalence.
frequency distribution
lists data values (either individually or by groups of intervals), along with their corresponding frequencies (or counts).
frequency table
a way of summarizing data; used as a record of how often each value (or set of values) of a variable occurs. A frequency table is used to summarize categorical, nominal, and ordinal data. It may also be used to summarize continuous data once the data is divided into categories.
F-test
A test for the equality of the variances of two populations having normal distributions, based on the ratio of the variances of a sample of observations taken from each. Most often encountered in the analysis of variance , where testing whether particular variances are the same also test for the equality of a set of means.
gold standard trials
A term usually retained for those clinical trials in which there is random allocation to treatments, a control group and double-blinding.
goodness of fit
Degree of agreement between an empirically observed distribution and a mathematical or theoretical distribution.
goodness-of-fit test
A statistical test of the hypothesis that data have been randomly sampled or generated from a population that follows a particular theoretical distribution or model. The most common such tests are chi-square tests.
hazard
Inherent capability of an agent or situation to have an adverse effect. A factor or exposure that may effect adversely effect health.
hazard rate (force of morbidity, instantaneous incidence rate)
A theoretical measure of the risk of an occurrence of an event, e.g. death or new disease, at a point in time, t , defined mathematically as the limit, as Δ t approaches zero, of the probability that an individual well at time t will experience the event by t + Δ t , divided by Δ t .
histogram
A graphical representation of a set of observations in which class frequencies are represented by the areas of rectangles centred on the class interval. If the latter are all equal, the heights of the rectangles are also proportional to the observed frequencies. A histogram of heights of elderly women is shown (see below).
historical controls
A group of patients treated in the past with a standard therapy, used as the control group for evaluating a new treatment on current patients. Although used fairly frequently in medical investigations, the approach is not to be recommended since possible biases, due to other factors that may have changed over the time, can never be satisfactory eliminated.
homogeneity (homogeneous)
A term that is used in statistics to indicate the equality of some quantity of interest (most often a variance), in a number of different groups, populations, etc.
homoscedasticity
homo means “same” and –scedastic means “scattered” therefore homoscedasticity means the constancy of the variance of a measure over the levels of the factors under study.
hypothesis testing
A general term for the procedure of assessing whether sample data is consistent or otherwise with statements made about the population.
incidence
A measure of the rate at which people without a disease develop the disease during a specific period of time. Calculated as
incidence = # new cases over a period of time/population at risk in the time period
it measures the appearance of disease. More generally, the number of new events, e.g. new cases of a disease in a specified population, within a specified period of time. The term incidence is sometimes wrongly used to denote incidence rate.
Independence
Two events are said to be independent if the occurrence of one is in no way predictable from the occurrence of the other. Two variables are said to be independent if the distribution of values of one is the same for all values of the other.
independent variable (explanatory variables)
The variables appearing on the right-hand side of the equations defining, for example, multiple regression or logistic regression, and which seek to predict or ‘explain’ the response variable. Using the term independent variable is not recommended since they are rarely independent of one another.
inference (statistical)
The process of drawing conclusions about a population on the basis of measurements or observations made on a sample of individuals for the population.
interaction
A term applied when two (or more) explanatory variables do not act independently on a response variable. The graphic below shows an example from a 2 x 2 factorial design. In statistics, interaction is also the necessity for a product term in a linear model.
intercept
The parameter in an equation derived from a regression analysis corresponding to the expected value of the response variable when all the explanatory variables are zero.
interquartile range
A measure of spread given by the difference between the first and third quartiles of a sample.
Inter-rater reliability (observer variation, inter-rater agreement, Concordance)
the degree of agreement among raters. It gives a score of how much homogeneity or consensus there is in the ratings given by judges. It is useful in refining the tools given to human judges, for example by determining if a particular scale is appropriate for measuring a particular variable. If various raters do not agree, either the scale is defective or the raters need to be re-trained. There are a number of statistics which can be used to determine inter-rater reliability. Different statistics are appropriate for different types of measurement. Some options are: joint-probability of agreement, Cohen’s kappa and the related Fleiss’ kappa, inter-rater correlation, concordance correlation coefficient and intra-class correlation.
intervention study
A study in which conditions are under the direct control of the investigator. In epidemiology, a study in which a population is selected for a planned trial of a regimen whose effects are measured by comparing the outcome of the regimen in the experimental group with the outcome of another regimen in a control group.
Kaplan-Meier estimate (product limit method)
A nonparametric method of compiling life or survival tables. This combines calculated probabilities of survival and estimates to allow for censored observations, which are assumed to occur randomly. The intervals are defined as ending each time an event (death, withdrawal) occurs and are therefore unequal.